SAN DIEGO, May 10, 2018 (GLOBE NEWSWIRE) -- Organovo Holdings ( ONVO ), Inc. (“Organovo”) today announced that it will present new preclinical data showing the strong functionality of its liver therapeutic tissue in a second IND-track program for Type 1 Tyrosinemia. This data will be presented at the World Advanced Therapies and Regenerative Medicine Congress in London on May 18, 2018 by Benjamin Shepherd, Ph.D., Senior Director of Therapeutics, at Organovo ( ONVO ).
Type 1 Tyrosinemia is a rare disease characterized by a patient’s inability to metabolize the amino acid tyrosine due to a deficiency of the enzyme fumarylacetoacetate hydrolase. This disorder frequently causes severe liver damage, with current treatment options often limited to organ transplantation. Organovo ( ONVO ) recently studied the potential benefit of its NovoTissues® in an established animal model for Type 1 Tyrosinemia, and showed retention and sustained functionality post-implamantation. Pathologic evaluation of the diseased animals receiving implanted bioprinted liver tissues also suggested an improvement in liver health and extended survival versus non-treated animals.
The Company will also summarize its ongoing progress in its first IND-track program for its NovoTissues treatment of alpha-1 antitrypsin deficiency (“A1AT”). This indication, which received orphan drug designation from the U.S. Food & Drug Administration in 2017, similarly represents a patient population that is in desperate need of new treatment options. Organovo ( ONVO ) previously disclosed the results of its preclinical studies in an established animal model for A1AT. Serum and histopathologic evaluation of the implanted therapeutic tissue showed engraftment, retention and a high degree of disease clearing through 125 days post-implantation. These results also demonstrated the sustained presence of key human liver proteins such as albumin and A1AT in the animal bloodstream. Importantly, pathologic evaluation of diseased animals receiving implanted bioprinted liver tissues suggested a significant reduction in the pathologic hallmarks of the disease in the damaged region of the liver adjacent to the NovoTissues transplant.
“We’re encouraged that our 3D bioprinted liver tissues continue to show retention and functionality in a range of animal disease models, including two disease areas where there is critical unmet need and a significant impact on patient outcomes because of the dire shortage of liver transplants,” said Dr. Benjamin Shepherd, senior director of therapeutics, Organovo. “In each case, our objective in implanting a healthy tissue patch is to restore function or offset the deficiency of a specific enzyme abnormality, with the ultimate goal of delaying or reducing the need for a transplant.”
“We are delighted with the early readouts of these complementary therapeutics programs,” said Taylor J. Crouch, CEO, Organovo. “The results in this second disease area within the orphan group of inborn errors of metabolism support the opportunity for a synergistic, second IND-track that we will likely pursue alongside our lead IND-track program for A1AT deficiency. There is great utility in deploying a single implanted tissue to treat multiple diseases, all of which are life-threatening and have few alternative treatment options.”
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